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Activity for Genes as Medicine

Topic
  • Biochemistry & Molecular Biology
  • Biotechnology
  • Genetics
  • Patterns of Inheritance
  • Genetic Disease
Resource Type
  • Activities
  • Film Activities
Level
High School — GeneralHigh School — AP/IBCollege
Favorited By
16 Users
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Description

This activity explores the content and research presented in the short film Genes as Medicine, which tells the story of how scientists succeeded in developing a gene therapy for a type of congenital blindness.

Gene therapy is an approach for treating inherited diseases by delivering corrective versions of genes to patients. Genes as Medicine tells the story of the successes and setbacks of two scientists working to develop a gene therapy for Leber congenital amaurosis, a type of childhood blindness. The film discusses how autosomal recessive conditions are inherited, how scientists use viruses to deliver human genes to cells, and how model organisms are used to test treatments before they are used in patients.

The “Student Handout” probes students’ understanding of the key concepts addressed in the film. The “Educator Materials” document provides suggested pause points in the film with questions for students, background information, and detailed discussion points; a list of related resources and references; and an answer key for the “Student Handout.”

Student Learning Targets
  • ​​​​​​Explain how gene therapy can be used to treat inherited conditions with known causal mutations.
  • Discuss how basic, translational, and clinical research contribute to the development of new medicines.
Details
Estimated Time
One 50-minute class period.
Key Terms

dog, gene therapy, Leber congenital amaurosis, viral delivery

Primary Literature

Acland, Gregory M., Gustavo D. Aguirre, Jharna Ray, Qi Zhang, Tomas S. Aleman, Artur V. Cideciyan, Susan E. Pearce-Kelling, et al. “Gene therapy restores vision in a canine model of childhood blindness.” Nature Genetics 28, 1 (2001): 92–95. https://doi.org/10.1038/ng0501-92.   

Russell, Stephen, Jean Bennett, Jennifer A. Wellman, Daniel C. Chung, Zi-Fan Yu, Amy Tillman, Janet Wittes, et al. “Efficacy and safety of voretigene neparvovec (AAV2- hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.” The Lancet 390, 10097 (2017): 849–860. https://doi.org/10.1016/S0140-6736(17)31868-8.

Terms of Use

Please see the Terms of Use for information on how this resource can be used.

Version History
Date Published 01.24.18
Date Updated 01.24.18
Curriculum Connections
NGSS (2013)

HS-LS1-1, HS-LS3-1, HS-LS3-3; SEP6

AP Biology (2019)

IST-1.K, IST-1.P, IST-2.E, IST-3.G, IST-4.A, IST-4.B; SP1

IB Biology (2016)

3.4, 3.5, B.4

Common Core (2010)

ELA.RST.9-12.2, ELA.WHST.9-12.9

Vision and Change (2009)

CC2, CC3; DP1

Materials
Student Handout (PDF) 807 KB
Educator Materials (PDF) 1 MB
Use This Resource With
Video Resource
Genes as Medicine
Related Science News
Gene therapy for rare form of blindness wins US approval
Officials remove special rules for gene therapy experiments
In a milestone year, gene therapy finds its place in medicine

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